April 23, 2018April 23, 2018
Krystal Biotech’s KB103 Receives Orphan Medicinal Product Designation in Europe

Krystal Biotech recently announced that the European Medicines Agency (EMA) has granted the company’s investigational therapy KB103 orphan medicinal product ... Read more
April 16, 2018April 16, 2018
$3.8M Defense Grant Awarded to Develop Stem Cell Therapies for EB, Wound Care

The U.S. Department of Defense awarded $3.8 million to a consortium working to develop stem cell-based therapies for patients ... Read more
April 13, 2018April 13, 2018
NIH Agency Pioneers Collaborative Research into Rare Diseases

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed ... Read more
April 11, 2018April 11, 2018
Researchers Identify a Blood Biomarker for Epidermolysis Bullosa-associated Squamous Cell Carcinoma

Using a simple blood sample may be sufficient to detect a biomarker for skin cancer in epidermolysis bullosa patients, a ... Read more
April 9, 2018April 9, 2018
Scientists Reduce Blistering in EBA Mice by Honing In on the Immune System

Scientists have found that using inhibitors on specific parts of the so-called complement system, part of the immune system ... Read more
April 4, 2018April 4, 2018
EBA Can Lead to Acute Renal Failure, Case Report Contends

Patients with epidermolysis bullosa acquisita sometimes can develop acute renal failure, according to a case report. The report, “Acute ... Read more
April 2, 2018April 2, 2018
SYK Protein a Key Regulator of Inflammatory Response in EBA Mouse Model, Study Finds

A protein called SYK is a major regulator of the inflammatory response carried out by myeloid cells in mouse ... Read more
March 28, 2018March 28, 2018
Krystal Files Application to Start Clinical Trial of Gene Therapy Candidate KB103

Krystal Biotech has submitted an investigational new drug application with the U.S. Food and Drug Administration (FDA) to start a Phase 1/2 ... Read more
March 26, 2018March 26, 2018
Fibroblast Skin Injections Show Promise in Healing Wounds in Dystrophic Epidermolysis Bullosa Patients, Study Finds

Injecting fibroblasts directly into a wound led to higher rates of healing and complete closure than other more common ... Read more
March 21, 2018March 21, 2018
Amryt Pharma Advances Lead Candidate AP101 to Global Phase 3 Trial for EB

Amryt Pharma’s lead investigative therapy AP101, developed to reduce the time it takes for skin wounds to close, is ... Read more
March 19, 2018March 19, 2018
Krystal Biotech to File IND Application for KB103 to Treat Dystrophic Epidermolysis Bullosa

Krystal Biotech plans to file an investigational new drug (IND) application for its lead therapy candidate KB103 for the treatment of ... Read more
March 16, 2018March 16, 2018
Genome Sequencing and Its Clinical Potential Focus of NYC Rare Disease Day Event

Genetic sequencing and the speed with which it can help diagnose a child’s disease — in addition to revealing the ... Read more

April 23, 2018April 23, 2018

Krystal Biotech’s KB103 Receives Orphan Medicinal Product Designation in Europe

Krystal Biotech recently announced that the European Medicines Agency (EMA) has granted the company’s investigational therapy KB103 orphan medicinal product designation for the treatment of dystrophic epidermolysis bullosa (DEB). KB103 is ... Read more

April 16, 2018April 16, 2018

$3.8M Defense Grant Awarded to Develop Stem Cell Therapies for EB, Wound Care

The U.S. Department of Defense awarded $3.8 million to a consortium working to develop stem cell-based therapies for patients with inherited skin diseases such as epidermolysis bullosa (EB) and for ... Read more

April 13, 2018April 13, 2018

NIH Agency Pioneers Collaborative Research into Rare Diseases

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. ... Read more

April 11, 2018April 11, 2018

Researchers Identify a Blood Biomarker for Epidermolysis Bullosa-associated Squamous Cell Carcinoma

Using a simple blood sample may be sufficient to detect a biomarker for skin cancer in epidermolysis bullosa patients, a new study suggests. This is because the cancer-specific variant of ... Read more

April 9, 2018April 9, 2018

Scientists Reduce Blistering in EBA Mice by Honing In on the Immune System

Scientists have found that using inhibitors on specific parts of the so-called complement system, part of the immune system that aids the functioning of antibodies, led to improvements in ... Read more

April 4, 2018April 4, 2018

EBA Can Lead to Acute Renal Failure, Case Report Contends

Patients with epidermolysis bullosa acquisita sometimes can develop acute renal failure, according to a case report. The report, “Acute renal failure in a patient with epidermolysis bullosa acquisita,” was ... Read more

April 2, 2018April 2, 2018

SYK Protein a Key Regulator of Inflammatory Response in EBA Mouse Model, Study Finds

A protein called SYK is a major regulator of the inflammatory response carried out by myeloid cells in mouse models of epidermolysis bullosa acquisita (EBA), showing potential for a ... Read more

March 28, 2018March 28, 2018

Krystal Files Application to Start Clinical Trial of Gene Therapy Candidate KB103

Krystal Biotech has submitted an investigational new drug application with the U.S. Food and Drug Administration (FDA) to start a Phase 1/2 clinical trial of its lead gene therapy candidate KB103 for ... Read more

March 26, 2018March 26, 2018

Fibroblast Skin Injections Show Promise in Healing Wounds in Dystrophic Epidermolysis Bullosa Patients, Study Finds

Injecting fibroblasts directly into a wound led to higher rates of healing and complete closure than other more common methods in patients with recessive dystrophic epidermolysis bullosa (RDEB), a ... Read more

March 21, 2018March 21, 2018

Amryt Pharma Advances Lead Candidate AP101 to Global Phase 3 Trial for EB

Amryt Pharma’s lead investigative therapy AP101, developed to reduce the time it takes for skin wounds to close, is recruiting patients with epidermolysis bullosa for a pivotal Phase 3 ... Read more